Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 ...

Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for patients with spinal muscular atrophy (SMA) and other severe and ...

（大山脚16日讯）6岁女童自出生后便被诊断患有罕见的脊髓性肌萎缩症（Spinal Muscular Atrophy Type 2，简称SMA Type ...

A 'fragile' girl's life has been transformed thanks to "caring and loving" staff at a hospice. Ellie, 12, from Salisbury, ...

Newborn with SMA gene mutation receives presymptomatic treatment at SAT Hospital, a rare step in managing rare diseases.

But now there is a ray of light. Lilah is scheduled for surgery on July 15 that will see a metal ring called a halo fastened ...

Liu walks with an unsteady gait due to muscular atrophy in lower legs, she affectionately calls herself Waiwai, meaning ...

Spinal Muscular Atrophy treatment is evolving fast, with gene therapy, oral drugs, and next-gen muscle enhancers offering new hope and long-term outcomes. With each breakthrough in SMA treatment, we ...

The baby from Ranaghat in Nadia is suffering from spinal muscular atrophy (SMA) Type I, a condition that causes progressive ...

A girl was diagnosed with a rare muscle -wasting condition after she stopped crawling as a baby. Ellie Soares, 11, seemed ...